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Deficiency diseases

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 Rare diseases

What is a rare disease?

A rare disease is any disease for which the number of affected patients is below a certain level (in Korea this amounts to 20,000 patients or less), or a disease for which no appropriate therapies or alternative drugs are available. Such diseases are hard to cure and require long-term treatment. Typically, rare diseases result from genetic deficiencies or mutations. According to the WHO, about 7,000 rare diseases have been reported worldwide; of these, over 6,000 are caused by monogenic mutations. The number of individuals suffering from such diseases is estimated at 25-30 million worldwide. About 200 drugs have been approved by US FDA; at present, the category receiving most attention is that containing drugs designed to treat inherited metabolic disorders, which result from a deficiency of the enzymes or coenzymes that are responsible for ensuring correct operation of metabolic pathways.

Such diseases include lysosomal storage disease (LSD), which is caused by a genetic deficiency in the enzymes required to degrade the metabolic products that accumulate in lysosomes, and hemophilia, which is caused by a deficiency of blood coagulation factors. In general, patients suffering from rare diseases have few treatment choices and the annual costs per patient can be as high as hundreds of millions of won; therefore, new products are urgently required.

R&D trends

Currently available therapies for rare diseases include diet-based treatments, bone-marrow transplantation, and enzyme replacement therapy (ERT). In addition, gene correction or gene replacement technologies,and cell-based therapies using gene-edited cells, are also being developed; however, these therapies are still in the developmental phase and none (apart from ERT) show promise. Even ERT has side effects, such as the induction of drug-specific antibodies, and shows low rates of delivery to the brain. For these reasons, researchers are attempting to develop advanced therapeutics, such as targeted agents that can pass through the blood brain barrier(BBB). Most of the drugs used to treat rare diseases are developed exclusively by global pharmaceutical companies.

Mogam conducts...

studying blood coagulation factors to develop drugs to treat hemophilia.More recently, we have begun to develop long-acting blood coagulation factors. We are also developing drugs to treat LSD, particularly mucopolysaccharidosis, which is caused by the accumulation of mucopolysaccharides (glycosaminoglycans). We are also conducting research into cell-based therapies, including cell/gene delivery systems for genetic correction, and stem cell-based drugs.